For this study, researchers randomly assigned patients with advanced HCC to receive oral cabozantinib or a placebo.

Patients with hepatocellular carcinoma (HCC) previously treated with sorafenib have significantly improved overall survival (OS) and progression-free survival (PFS) rates when treated with cabozantinib compared with placebo, according to data being presented at the 2018 Gastrointestinal Cancers Symposium in San Francisco, California.

For the double-blind phase 3 CELESTIAL study (ClinicalTrials.gov Identifier: NCT01908426), researchers randomly assigned 707 patients with advanced HCC to receive oral cabozantinib 60 mg once daily or placebo; 470 and 237 patients were assigned to the cabozantinib and placebo arms, respectively. Eligible patients were previously treated with sorafenib, had disease progression after no more than 2 lines of systemic therapy, and were unamenable to curative therapy.

At the time of data analysis, 484 (68.4%) enrolled patients had died (317 [67.4%] patients in the cabozantinib arm and 167 [70.4%] patients in the placebo arm).

The most frequently reported grade 3 to 4 adverse events — which occurred at a higher rate in the cabozantinib arm — included hand-foot-skin reactions, hypertension, elevated aspartate aminotransferase, fatigue, and diarrhea.

The authors concluded that “[cabozantinib] significantly improved OS and PFS vs [placebo] in previously treated patients with advanced HCC. Adverse events were consistent with the known safety profile of [cabozantinib].”